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X-linked hypophosphatemia (XLH) is the most common form of heritable rickets. Current treatments include active vitamin D metabolites (e.g. calcitriol) and phosphate salts. There is no consistent weight-based dosing of calcitriol and phosphate now. The primary objective of this study is to establish the efficacy of different dose of calcitriol combined with neutral phosphate in children with XLH.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| High-dose | Experimental | Receiving 60 ng/kg/day of calcitriol and 30 mg/kg/day of elemental phosphorus. |
|
| Low-dose | Experimental | Receiving 20 ng/kg/day of calcitriol and 30 mg/kg/day of elemental phosphorus. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Calcitriol | Drug |
|
|
| Measure | Description | Time Frame |
|---|---|---|
| Change From Baseline to Post-treatment in Severity of Rickets as Measured by Rickets Severity Score (RSS) Total Score | RSS range from 0 to 10, and higher RSS represent severer rickets. It is consisted of score of worst wrist (0-4) and worst knee (0-6). WRIST-score both radius and ulna separately-2 bones × 2 points = 4 points possible KNEE-score both femur and tibia separately Multiply the grade in A by the multiplier in B for each bone, then add femur and tibia scores together A: Grade 1 2 3 B: Multiplier Portion of growth plate affected 0.5 ≤ 1 condyle or plateau
| Baseline, Month 12, 24 |
| Measure | Description | Time Frame |
|---|---|---|
| Changes From Baseline to Post-treatment in Severity of Rickets as Measured by RSS Wrist and Knee Score | RSS range from 0 to 10, and higher RSS represent severer rickets. It is consisted of score of worst wrist (0-4) and worst knee (0-6). WRIST-score both radius and ulna separately-2 bones × 2 points = 4 points possible KNEE-score both femur and tibia separately Multiply the grade in A by the multiplier in B for each bone, then add femur and tibia scores together A: Grade 1 2 3 B: Multiplier Portion of growth plate affected 0.5 ≤ 1 condyle or plateau
|
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| Weibo Xia, MD | Contact | +86 13501002126 | xiaweibo8301@163.com |
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Department of Endocrinology, Peking Union Medical College Hospital | Recruiting | Beijing | Beijing Municipality | 100730 | China |
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| ID | Term |
|---|---|
| D053098 | Familial Hypophosphatemic Rickets |
| ID | Term |
|---|---|
| D063730 | Rickets, Hypophosphatemic |
| D012279 | Rickets |
| D001851 | Bone Diseases, Metabolic |
| D001847 | Bone Diseases |
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| ID | Term |
|---|---|
| D002117 | Calcitriol |
| ID | Term |
|---|---|
| D004100 | Dihydroxycholecalciferols |
| D006887 | Hydroxycholecalciferols |
| D002762 | Cholecalciferol |
| D002782 | Cholestenes |
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| Baseline, Month 12, 24 |
| Changes From Baseline to Post-treatment in Growth Velocity | Changes From Baseline to Post-treatment in Severity of Rickets as Measured by RSS Wrist and Knee Score | Baseline, Month 12, 24 |
| Changes From Baseline to Post-treatment in Serum Total Alkaline Phosphatase (TALP) Levels | Changes From Baseline to Post-treatment in serum ALP | Baseline, Month 3, 6, 12, 18, 24 |
| Changes From Baseline to Post-treatment in serum Carboxy-terminal Collagen Crosslinks (CTX) Levels | Changes From Baseline to Post-treatment in serum CTX | Baseline, Month 3, 6, 12, 18, 24 |
| Changes From Baseline to Post-treatment in Serum Phosphorus Levels | Changes From Baseline to Post-treatment in Serum Phosphorus Levels | Baseline, Month 3, 6, 12, 18, 24 |
| Changes From Baseline to Post-treatment in Severity of Dental Abscess | The frequency and number of dental abscess will be collected by self-report and examed by investigators | Baseline, Month 3, 6, 12, 18, 24 |
| Changes From Baseline to Post-treatment in Severity of Bone Pain as Measured by Visual Analog Pain Scales | Visual Analog Pain Scales (VAS) range from 0-10, and higher score represents severer pain | Baseline, Month 3, 6, 12, 18, 24 |
| Changes From Baseline to Post-treatment in Severity of Leg Deformities | The longest distance between two sides of thighs, knees, shanks and ankles are measured to evaluate the severity of leg deformities. | Baseline, Month 3, 6, 12, 18, 24 |
| Changes From Baseline to Post-treatment in Serum Osteocalcin Levels | Changes From Baseline to Post-treatment in Serum Osteocalcin Levels | Baseline, Month 3, 6, 12, 18, 24 |
| Changes From Baseline to Post-treatment in Height | Changes From Baseline to Post-treatment in Height | Baseline, Month 3, 6, 12, 18, 24 |
| Changes From Baseline to Post-treatment in Quality of Life as Measured by Patient-reported Outcomes Measurement Information System | Changes From Baseline to Post-treatment in Quality of Life as Measured by Patient-reported Outcomes Measurement Information System | Baseline, Month 3, 6, 12, 18, 24 |
| D009140 |
| Musculoskeletal Diseases |
| D007015 | Hypophosphatemia, Familial |
| D015499 | Renal Tubular Transport, Inborn Errors |
| D007674 | Kidney Diseases |
| D014570 | Urologic Diseases |
| D052776 | Female Urogenital Diseases |
| D005261 | Female Urogenital Diseases and Pregnancy Complications |
| D000091642 | Urogenital Diseases |
| D052801 | Male Urogenital Diseases |
| D008664 | Metal Metabolism, Inborn Errors |
| D008661 | Metabolism, Inborn Errors |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D008659 | Metabolic Diseases |
| D009750 | Nutritional and Metabolic Diseases |
| D002128 | Calcium Metabolism Disorders |
| D017674 | Hypophosphatemia |
| D010760 | Phosphorus Metabolism Disorders |
| D014808 | Vitamin D Deficiency |
| D001361 | Avitaminosis |
| D003677 | Deficiency Diseases |
| D044342 | Malnutrition |
| D009748 | Nutrition Disorders |
| D002776 |
| Cholestanes |
| D013256 | Steroids |
| D000072473 | Fused-Ring Compounds |
| D011083 | Polycyclic Compounds |
| D013261 | Sterols |
| D014807 | Vitamin D |
| D012632 | Secosteroids |
| D008563 | Membrane Lipids |
| D008055 | Lipids |